Auburn University and the University of Massachusetts Medical School team with French Biotech Company to treat neurologic disorder in children

April 06, 2015 @ 4:46 p.m.

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Auburn University and the University of Massachusetts Medical (UMMS) school have partnered with Paris-based biotech firm, Lysogene, in studies related to GM1 glandliosidosis.

GM1 gangliosidosis is a member of a group of about 40 related disorders that result from dysfunction of lysosomal enzymes or related proteins. GM1 is a neurologic condition of children that is typically fatal by two years of age. An inherited disorder, GM1 progressively destroys nerve cells in the brain and spinal cord, and is a neurodegenerative disease much like Parkinson’s or Alzheimer’s. There is no cure or effective treatment available and it affects one in every 100,000 to 200,000 newborns.

For more than 40 years, Scott Richey Research Center (SRRC) in Auburn University’s College of Veterinary Medicine has researched GM1 gangliosidosis in felines, as GM1 also occurs naturally in cats. The goal of SRRC’s GM1 research is to find a cure for feline GM1 and, through research partners, successfully apply similar therapies for application in humans.

One of the most successful experimental treatments found to date is gene therapy, using a non-harmful viral vector (AAV) to produce missing enzymes, in this case beta-galactosidase. In feline trials at SRRC, it has successfully restored beta-galactosidase levels to normal, prevented storage of GM1 ganglioside, and quadrupled the lifespan of GM1 cats as survival benefits continue to increase.

Auburn University, Lysogene, and the UMMS will work together on preclinical studies related to AAV gene therapy. According to a press release from Lysogene, the collaboration will combine Lysogene's outstanding translational and clinic expertise in gene therapy for central nervous system disorders with the unique preclinical expertise and infrastructure of UMMS and with Auburn's ability to design and text innovative AAV-based gene therapy approaches to treatment.